News item

Archive of: 2018

The SHCA has published two reports analysing the current system in England for determining access to treatment for patients with rare and complex conditions.

Achieving fairness for all highlights the complexity of appraisal mechanisms for rare disease treatments, involving multiple different methodologies, processes and timelines. For patients and representatives, this complexity creates significant challenges in being able to fully engage with national decision-takers to inform their evaluations. This challenge is compounded by the methodologies used by these national bodies, which do not provide sufficient flexibility to take into account the characteristics common to rare disease treatments of less extensive clinical evidence and relative higher per-patient costs than treatments for more common conditions.

The SHCA is therefore calling for reform to the system, to address imbalances in the assessment of rare disease treatments. The SHCA has developed a set of principles that should guide national policymakers in delivering this reform:

There must be a ‘premium for rarity’ – to enable the system to fairly account for the higher cost of treatments for rare diseases

In the event of uncertainty, the presumption should be in favour of access – to enable the system to fairly account for the uncertainty in the evidence base for new treatments, as in the case of the Cancer Drugs Fund

The patient voice must be heard – to enable smaller charities to fully engage in processes and inform decision-taking

Decisions must be made transparently – to ensure that the system has the confidence of patients

The NHS must work with pharmaceutical companies to enable it to take consistent decisions over time – to provide sustainability of funding for treatments for rare diseases

A Backwards Step? provides an assessment of the April 2017 changes to NICE’s processes and methods, when a ‘Budget Impact Test’ (BIT) for all treatments was created and a cost-effectiveness threshold was introduced to the Highly Specialised Technologies (HST) programme for the first time. The report was developed in response to concerns raised by SHCA members about the changes. In a survey we carried out in April and May this year, 81% of respondents reported that they were either very concerned (47%) or concerned (34%) about the potential impact the reforms could have on patients with rare and complex conditions.

More than a year on from the changes, the SHCA sought to assess the extent to which these concerns had been realised. Frustratingly, we encountered challenges in carrying out a comprehensive assessment, due to low levels of transparency in the case of the BIT and extended delays in the publication of HST guidance. However, it does appear that the new HST thresholds are impeding patients’ access to treatment.

The SHCA is therefore calling for the scheduled review of the BIT and HST thresholds to be brought forward and for patients and their representatives to be fully engaged in this process. In the interim period, we believe that the thresholds should be lifted and that NICE should work with NHS England to take steps to improve transparency around the reporting of the application of the BIT.